Introduction

The drug ravulizumab (trade name: Ultomiris) has been approved in Germany since July 2019 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults.

PNH is a very rare acquired (not inherited) disease of the hematopoietic stem cells (HSCs). It is caused by a mutated (changed) gene. As a result of this gene mutation, the body’s immune system destroys some of the red blood cells. When the cells are destroyed, substances responsible for the red color of blood are released and can change the color of the urine. PNH may also cause the following symptoms:

  • Anemia from abnormal destruction of red blood cells (hemolysis)
  • Higher risk of having blood clots (thrombosis)
  • Cytopenia: Lower blood cell count

The signs of anemia may include shortness of breath and breathlessness during physical exertion. People most commonly develop PNH between the ages of 25 and 45.

Ravulizumab is an antibody that is believed to help prevent the immune system from attacking the red blood cells.

Application

Ravulizumab is given as a drip directly into the bloodstream through a vein (an intravenous infusion). The dose will depend on the patient's body weight. After ravulizumab is used for the first time, the infusion is given again 2 weeks later, and every 8 weeks after that. Each infusion takes about 2.5 hours at most. The infusions are continued as a life-long treatment.

Other treatments

So far there is only one other medication for PNH in adults. It contains the antibody eculizumab.

Assessment

In 2019, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) looked into the advantages and disadvantages of ravulizumab when compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in adults.

The manufacturer provided suitable data for the following groups of patients:

  • People with a high level of PNH disease activity who have not had previous treatment
  • People with clinically stable PNH who have already had treatment with eculizumab for at least six months

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More information

This information summarizes the main results of a review produced by the Institute for Quality and Efficiency in Health Care (IQWiG, Germany). The review was commissioned by the German Federal Joint Committee (G-BA) as part of the “early benefit assessment of medications.” On the basis of the review and the hearings received, the G-BA passed a resolution on the added benefit of ravulizumab (Ultomiris).